Apertura Joins ARPA-H Consortium to Target Rare Pediatric Epilepsy

The consortium will utilize Apertura’s proprietary TfR1 CapX, an engineered adeno-associated virus capsid capable of crossing the blood-brain barrier via intravenous administration. By targeting the human transferrin receptor 1, the technology enables precise delivery to the brain and spinal cord, overcoming a major hurdle in treating neurogenetic diseases. This platform will be integrated with advanced base and prime gene editing tools developed in the laboratory of David Liu at the Broad Institute.

This collaborative effort, dubbed the Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform, seeks to consolidate manufacturing processes and regulatory pathways to shorten development timelines. Andrew Steinsapir, acting CTO at Apertura, noted that traditional approaches struggle to address the diversity of the more than 400 genes linked to developmental and epileptic encephalopathies. By pooling infrastructure with patient advocates and contract manufacturer Viralgen, the group intends to move beyond isolated research efforts.

Apertura’s role in this consortium aligns with its broader strategy to license the TfR1 CapX technology across the biotechnology sector. With multiple licensing agreements already in place, the company expects several programs utilizing this capsid to begin clinical trials within the next 12 months. This project represents a significant expansion of the technology’s utility in clinical settings, following earlier research published in Science regarding the company's foundational capsid engineering.